AVEO Pharmaceuticals, Inc. Receives Orphan Medicinal Product Designation for Tivozanib for the Treatment of Renal Cell Carcinoma by the European Medicines Agency

CAMBRIDGE, Mass., Jun 24, 2010 (BUSINESS WIRE) --AVEO Pharmaceuticals, Inc. (NASDAQ: AVEO), a biopharmaceutical company focused on discovering, developing and commercializing cancer therapeutics, today announced it has received orphan medicinal product designation for tivozanib (N-{2-chloro-4-[(6,7-dimethoxy-4-quinolyl)oxy]phenyl}-N'-(5-methyl-3-isoxazolyl) urea hydrochloride monohydrate), its oral, triple VEGF receptor inhibitor, for the treatment of renal cell carcinoma by the European Medicines Agency (EMA). According to the EMA, tivozanib was awarded orphan medicinal product designation based on the prevalence of renal cell carcinoma among people in the European Union (EU); the life-threatening nature of the disease particularly for those with advanced or metastatic renal cell carcinoma; and the assumption that tivozanib may provide significant benefit for patients with renal cell carcinoma, and may be more potent and specific than existing treatments with similar mechanism of action as supported by preliminary clinical results.

"Following the positive results from our Phase 2 clinical trial of tivozanib, we are very pleased to receive the EMA's orphan medicinal product designation for tivozanib, which represents an important milestone as we continue with the clinical development of tivozanib in renal cell cancer (RCC)," said Tuan Ha-Ngoc, president and chief executive officer of AVEO. "We are conducting a number of clinical trials of tivozanib both as monotherapy and in combination with other anti-cancer agents, and we believe tivozanib may represent an important and differentiated new therapeutic option to treat RCC."

Orphan medicinal product designation is awarded by the EMA for drugs that are shown to diagnose, prevent or treat life-threatening or rare, serious conditions that affect fewer than five in 10,000 people in the EU. Companies that receive orphan medicinal product designation by the EMA receive, among other benefits, market exclusivity in the EU for ten years following market authorization, protocol assistance, reduced fees and the opportunity to receive grants from the EU and member states supporting research and development. Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. Demonstration of quality, safety and efficacy is necessary before a designated orphan medicinal product can be granted a marketing authorization.

In February 2010, AVEO initiated patient enrollment in the TIVO-1 trial, a global Phase 3 clinical trial, for tivozanib in advanced RCC. TIVO-1 is a randomized, controlled trial evaluating tivozanib compared to sorafenib (Nexavar(R)). Sorafenib is approved by both the U.S. Food and Drug Administration (FDA) and the EMA for use in patients with advanced RCC. The primary endpoint of the trial is to compare the progression-free survival (PFS) of tivozanib vs. sorafenib. Secondary endpoints include overall survival, objective response rate, duration of response and quality of life. Independent radiology review will be conducted for all CT scans and radiographs. Patients with RCC of clear cell histology that have had a prior nephrectomy and that have not received prior VEGF-targeted therapy are eligible for this trial. Patients will be randomized 1:1 to tivozanib or sorafenib. Patients who demonstrate disease progression during treatment with sorafenib will have the opportunity to be treated with tivozanib by participating in a separate long-term treatment protocol. Additional information on the trial design can be found online at www.clinicaltrials.gov.

Prior to launching TIVO-1, AVEO successfully completed a 272-patient Phase 2 clinical trial of tivozanib in patients with advanced RCC. In March, AVEO presented an updated analysis of the results from its Phase 2 clinical trial of tivozanib at the 2010 Genitourinary Cancers Symposium, that reported the median PFS achieved by patients with advanced clear cell RCC who had undergone a prior nephrectomy was 14.8 months - comparing favorably to historical data from trials testing other currently approved VEGF receptor inhibitors in RCC.

About Tivozanib

Tivozanib, an investigational new drug, is a highly potent and selective inhibitor of VEGF receptors 1, 2 and 3, exhibiting picomolar inhibitory activity against all three receptors. Due to its potency and specificity, AVEO believes tivozanib may enable optimal inhibition of the VEGF pathway, while minimizing side effects associated with off-target activity. Such a profile may enable tivozanib to be more readily combined with standard chemotherapy as well as other targeted therapies, potentially increasing the breadth of its clinical utility.

Following the successful completion of Phase 1 and Phase 2 clinical trials, AVEO has launched a comprehensive clinical development program in support of tivozanib. In addition to the TIVO-1 trial, AVEO is currently conducting multiple Phase 1b clinical trials of tivozanib in various combinations and dosing regimens in RCC and additional solid tumor indications, including breast cancer and colorectal cancer.

About AVEO

AVEO Pharmaceuticals (NASDAQ: AVEO) integrates a proprietary cancer biology platform with drug development and commercial expertise in its efforts to discover and develop targeted cancer therapeutics. The company's lead product, tivozanib, is an oral, triple VEGF receptor inhibitor with potential for a best-in-class profile. Tivozanib is currently being investigated in a global, randomized Phase 3 clinical trial called TIVO-1 comparing tivozanib to sorafenib in advanced kidney cancer, as well as additional clinical studies in other solid tumor types. AVEO's proprietary, integrated cancer biology platform offers the company a unique advantage in oncology drug development that both increases the probability of clinical success and provides a discovery engine for high-value targets. This approach has resulted in a promising pipeline of monoclonal antibodies against novel targets including HGF, ErbB3, RON, Notch and FGFR. For more information, please visit the company's website at www.aveopharma.com.

Any statements in this press release about our future expectations, plans and prospects, including statements about tivozanib's ability to provide significant benefit for patients with RCC and be an important and differentiated new therapeutic option to treat RCC, tivozanib's potency and tolerability as compared to existing treatments for RCC,tivozanib's potential as an important and highly differentiated new therapeutic option to treat renal cell cancer,the company's cancer biology platform increasing the probability of clinical success and providing a discovery engine for high-value targets, and other statements containing the words "believes," "anticipates," "plans," "expects," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks relating to: our ability to successfully research, develop and obtain and maintain regulatory approvals for tivozanib and our other product candidates, including our ability to obtain and maintain market authorization for tivozanib in the EU; the possibility that positive results in our Phase 2 clinical trial of tivozanib may not be predictive of the results in our Phase 3 clinical trial; our inability to obtain and maintain adequate protection for intellectual property rights relating to our product candidates and technologies; unplanned operating expenses and our inability to raise substantial additional funds to achieve our goals; general economic and industry conditions; and other factors discussed in the "Risk Factors" section of our most recent Form 10-Q filed with the Securities and Exchange Commission, and in other filings that we periodically make with the SEC. In addition, the forward-looking statements included in this press release represent our views as of the date of this press release. We anticipate that subsequent events and developments will cause our views to change. However, while we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.